EP3624858A4 - High activity regulatory elements - Google Patents

High activity regulatory elements Download PDF

Info

Publication number
EP3624858A4
EP3624858A4 EP18803184.3A EP18803184A EP3624858A4 EP 3624858 A4 EP3624858 A4 EP 3624858A4 EP 18803184 A EP18803184 A EP 18803184A EP 3624858 A4 EP3624858 A4 EP 3624858A4
Authority
EP
European Patent Office
Prior art keywords
regulatory elements
high activity
activity regulatory
elements
regulatory
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP18803184.3A
Other languages
German (de)
French (fr)
Other versions
EP3624858A1 (en
Inventor
Kartik RAMAMOORTHI
Stephanie TAGLIATELA
Anne TANENHAUS
Andrew Young
Szu-Ying Chen
Chi Zhang
Stephanie Martin
David OBERKOFLER
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Encoded Therapeutics Inc
Original Assignee
Encoded Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Encoded Therapeutics Inc filed Critical Encoded Therapeutics Inc
Publication of EP3624858A1 publication Critical patent/EP3624858A1/en
Publication of EP3624858A4 publication Critical patent/EP3624858A4/en
Pending legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0381—Animal model for diseases of the hematopoietic system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Obesity (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
EP18803184.3A 2017-05-19 2018-05-18 High activity regulatory elements Pending EP3624858A4 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762508968P 2017-05-19 2017-05-19
PCT/US2018/033515 WO2018213786A1 (en) 2017-05-19 2018-05-18 High activity regulatory elements

Publications (2)

Publication Number Publication Date
EP3624858A1 EP3624858A1 (en) 2020-03-25
EP3624858A4 true EP3624858A4 (en) 2021-06-23

Family

ID=64274721

Family Applications (1)

Application Number Title Priority Date Filing Date
EP18803184.3A Pending EP3624858A4 (en) 2017-05-19 2018-05-18 High activity regulatory elements

Country Status (11)

Country Link
US (1) US12104175B2 (en)
EP (1) EP3624858A4 (en)
JP (3) JP7315475B2 (en)
KR (2) KR20240024326A (en)
CN (1) CN110944674B (en)
AU (1) AU2018269050A1 (en)
BR (1) BR112019024222A2 (en)
CA (1) CA3063464A1 (en)
MA (1) MA49153A (en)
TW (1) TWI811219B (en)
WO (1) WO2018213786A1 (en)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CN110944674B (en) 2017-05-19 2024-07-19 编码治疗公司 High activity regulatory element
MA50942A (en) 2017-12-01 2020-10-07 Encoded Therapeutics Inc MODIFIED DNA BINDING PROTEINS
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
EP3914719A4 (en) * 2019-01-24 2023-04-05 Valorisation-HSJ, Limited Partnership Cell-specific transcriptional regulatory sequences and uses thereof
EA202192801A1 (en) * 2019-04-12 2022-02-24 Инкоудид Терапьютикс, Инк. COMPOSITIONS AND METHODS OF DRUG ADMINISTRATION
JP2022542839A (en) 2019-07-19 2022-10-07 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー Recombinase compositions and methods of use
CN115151648A (en) * 2020-02-14 2022-10-04 阿尔特拉吉尼克斯制药公司 Gene therapy for the treatment of CDKL5 deficient disorders
WO2023015304A1 (en) * 2021-08-05 2023-02-09 Insmed Incorporated Adeno-associated virus particles and methods of use thereof
WO2024079661A1 (en) * 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Atp7b gene therapy
CN117925624B (en) * 2024-03-22 2024-05-31 上海凌医生物科技有限公司 Metal response regulating element
CN117947035A (en) * 2024-03-25 2024-04-30 上海凌医生物科技有限公司 Promoter for enhancing gene expression

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001036620A2 (en) * 1999-11-16 2001-05-25 Genzyme Corporation Vectors and transgenies with regulatory elements for gene delivery to the liver
WO2013033627A2 (en) * 2011-09-01 2013-03-07 The Regents Of The University Of California Diagnosis and treatment of arthritis using epigenetics

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6436703B1 (en) * 2000-03-31 2002-08-20 Hyseq, Inc. Nucleic acids and polypeptides
EP1783645A1 (en) * 2005-11-08 2007-05-09 Actigenics Methods for the identification of microRNA and their applications in research and human health
EP2097538A4 (en) * 2006-12-07 2011-11-30 Switchgear Genomics Transcriptional regulatory elements of biological pathways, tools, and methods
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
ES2752191T3 (en) * 2012-02-17 2020-04-03 Childrens Hospital Philadelphia Compositions and methods with AAV vectors for gene transfer to cells, organs and tissues
CN104884627A (en) 2012-10-25 2015-09-02 托卡根公司 Retroviral vector with mini-promoter cassette
KR102526616B1 (en) * 2014-12-17 2023-04-27 푼다시온 파라 라 인베스티가시온 메디카 아플리카다 Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson disease
TWI707951B (en) * 2015-04-08 2020-10-21 美商健臻公司 Production of oversized adeno-associated vectors
CN110944674B (en) 2017-05-19 2024-07-19 编码治疗公司 High activity regulatory element
MA50942A (en) * 2017-12-01 2020-10-07 Encoded Therapeutics Inc MODIFIED DNA BINDING PROTEINS
AU2020229382A1 (en) * 2019-02-28 2021-09-16 Encoded Therapeutics, Inc. Compositions and methods for treating laminopathies

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001036620A2 (en) * 1999-11-16 2001-05-25 Genzyme Corporation Vectors and transgenies with regulatory elements for gene delivery to the liver
WO2013033627A2 (en) * 2011-09-01 2013-03-07 The Regents Of The University Of California Diagnosis and treatment of arthritis using epigenetics

Also Published As

Publication number Publication date
KR102636351B1 (en) 2024-02-15
KR20240024326A (en) 2024-02-23
JP2020520643A (en) 2020-07-16
BR112019024222A2 (en) 2020-08-18
US20200231943A1 (en) 2020-07-23
AU2018269050A1 (en) 2020-01-16
EP3624858A1 (en) 2020-03-25
TW201903152A (en) 2019-01-16
TWI811219B (en) 2023-08-11
US12104175B2 (en) 2024-10-01
JP2022173348A (en) 2022-11-18
MA49153A (en) 2021-03-24
CN110944674B (en) 2024-07-19
JP2024015408A (en) 2024-02-01
KR20200026814A (en) 2020-03-11
CA3063464A1 (en) 2018-11-22
CN110944674A (en) 2020-03-31
WO2018213786A1 (en) 2018-11-22
JP7315475B2 (en) 2023-07-26

Similar Documents

Publication Publication Date Title
EP3624858A4 (en) High activity regulatory elements
EP3692489A4 (en) Quantumproof blockchain
EP3350734A4 (en) Activity capability monitoring
EP3240409A4 (en) Picolinamides with fungicidal activity
EP3244843A4 (en) Stent
EP3373776A4 (en) Egg poacher
EP3626813A4 (en) Incubator
EP3330256A4 (en) HETEROCYCLIC DERIVATIVE HAVING TrkA-INHIBITING ACTIVITY
EP3257482A4 (en) Stent
EP3359556A4 (en) Modulating gamma - c -cytokine activity
EP3688700A4 (en) Message-credentialed blockchains
EP3265567A4 (en) Regulatory elements from labyrinthulomycetes microorganisms
EP3684172A4 (en) Genome-edited birds
EP3279323A4 (en) Amadoriase having improved specific activity
EP3680012A4 (en) Reactor
EP3503913A4 (en) Staphtame activity on biofilms
EP3295505A4 (en) Nitrogen-functionalized platinum-iridium electrocatalyst
EP3718568A4 (en) Activity modulator
EP3607339A4 (en) Activity mri
EP3585947A4 (en) Embanking arrangement
EP3486982A4 (en) Structure
EP3357464A4 (en) Underpants-type diaper
EP3685644B8 (en) Rfid-carrel
AU2017904885A0 (en) Catalyst
AU2017904922A0 (en) Shielding Structure

Legal Events

Date Code Title Description
STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE

PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE

17P Request for examination filed

Effective date: 20191209

AK Designated contracting states

Kind code of ref document: A1

Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

AX Request for extension of the european patent

Extension state: BA ME

REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40025397

Country of ref document: HK

RIC1 Information provided on ipc code assigned before grant

Ipc: C12N 15/861 20060101ALI20210216BHEP

Ipc: C12N 15/85 20060101ALI20210216BHEP

Ipc: A61K 48/00 20060101AFI20210216BHEP

A4 Supplementary search report drawn up and despatched

Effective date: 20210526

RIC1 Information provided on ipc code assigned before grant

Ipc: A61K 48/00 20060101AFI20210519BHEP

Ipc: C12N 15/85 20060101ALI20210519BHEP

Ipc: C12N 15/861 20060101ALI20210519BHEP

P01 Opt-out of the competence of the unified patent court (upc) registered

Effective date: 20230527